290 patents
Page 9 of 15
Utility
Compositions and Methods for Treating Pompe Disease
12 Aug 21
The present application provides methods of treating Pompe disease such as infantile-onset Pompe disease (IOPD) using a pharmaceutical composition comprising an oligosaccharide-acid α-glucosidase (GAA) conjugate, such as avalglucosidase alfa.
Kristina AN HAACK, Patrick FINN
Filed: 8 Feb 21
Utility
AAV virions with decreased immunoreactivity and uses therefor
10 Aug 21
Methods of making and using recombinant AAV virions with decreased immunoreactivity are described.
Alejandra E. Arbetman, Peter C. Colosi, Michael A. Lochrie, Richard T. Surosky
Filed: 29 Jan 19
Utility
Fusion proteins comprising PDGF and VEGF binding portions and methods of using thereof
10 Aug 21
The present provides fusion proteins comprising PDGF and VEGF binding portions, and recombinant viral particles encoding the fusion proteins.
Peter Pechan, Jeffery Ardinger, Hillard Rubin, Samuel Wadsworth, Abraham Scaria
Filed: 20 Nov 18
Utility
Intraventricular Enzyme Delivery for Lysosomal Storage Diseases
29 Jul 21
Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease.
James DODGE, Marco A. PASSINI, Lamya SHIHABUDDIN, Seng H. CHENG
Filed: 29 Dec 20
Utility
Methods for the quantitation of polypeptides
20 Jul 21
Provided are methods for quantitating an amount of a polypeptide that comprises a portion of an antibody present in a sample (e.g., a plasma or serum sample) wherein the antibody comprises a constant region (e.g., a heavy chain or light chain constant region) that comprises an engineered mutation.
Christopher Morgan, Xiaokui Zhang
Filed: 11 Jan 19
Utility
Perfusion culturing methods and uses thereof
13 Jul 21
Provided herein are methods of culturing a mammalian cell and various methods that utilize these culturing methods.
Agata Villiger-Oberbek, Jianguo Yang, Yang Yang, Gabrielle Lorenzo
Filed: 5 Jun 15
Utility
Aav Vectors for Retinal and CNS Gene Therapy
24 Jun 21
Provided herein are improved rAAV (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of ocular disorders or CNS disorders wherein the rAAV comprise one or more substitutions of amino acids that interact with heparan sulfate proteoglycan.
Abraham SCARIA, Jennifer SULLIVAN, Lisa M. STANEK, Lamya S. SHIHABUDDIN
Filed: 5 Mar 21
Utility
Methods of Processing a Fluid Including a Recombinant Therapeutic Protein and Use Thereof
10 Jun 21
Provided herein are methods of processing a fluid that include the use of one or both of a circuit system including a tangential flow filtration (TFF) unit and a circuit system including a tangential flow virus filtration (TFVF) unit.
Tarl Vetter, Michael Coolbaugh, Veena Warikoo, Konstantin Konstantinov
Filed: 19 Feb 21
Utility
Tropomyosin-Related Kinase (TRK) Inhibitors
10 Jun 21
Tropomyosin-related kinase inhibitors (Trk inhibitors) are small molecule compounds useful in the treatment of disease.
John L. Kane, JR., Gloria Matthews, Markus Metz, Michael Kothe, Jinyu Liu, Andrew Scholte
Filed: 25 Sep 20
Utility
Synthesis of Udp-glucose: N-acylsphingosine Glucosyltransferase Inhibitors
10 Jun 21
Disclosed is a novel enantiomeric synthesis ceramide-like inhibitors of UDP-glucose:N-acylsphingosine glucosyltransferase.
Bradford H. HIRTH, Craig SIEGEL
Filed: 23 Jul 20
Utility
Inhibitors of T-cell activation
8 Jun 21
The present invention provides a bispecific biologic comprising a ligand specific for CTLA-4 and a ligand specific for a pMHC complex.
Yunxiang Zhu, Jozsef Karman, Ronnie Wei, Canwen Jiang, Seng Cheng
Filed: 19 Oct 17
Utility
Bone-Targeting Antibodies
3 Jun 21
Provided are recombinant and chemically-conjugated antibodies and fragments thereof modified with one or more poly-aspartate (poly-D) peptides (e.g., a D10 sequence) to improve localization of the antibodies or fragments to bone.
Huawei Qiu, Sunghae Park, James Stefano
Filed: 23 Oct 20
Utility
Marker for Acid Sphingomyelinase Disorders and Uses Thereof
3 Jun 21
The present disclosure provides methods of screening, diagnosing, monitoring and/or treating acid sphingomyelinase (ASM) disorders such as Niemann-Pick disease.
Wei-Lien Chuang, Gerald F. Cox, X. Kate Zhang
Filed: 11 Dec 20
Utility
Use of CXCR4 Antagonists
3 Jun 21
Presently disclosed are methods and compositions for treating or preventing WHIM syndrome and certain other disorders or conditions with a certain CXCR4 antagonist.
David C. DALE, Gary J. BRIDGER, Frank J. HSU
Filed: 13 Jul 20
Utility
Pharmaceutical Compositions for Treating Acid Sphingomyelinase Deficiency
20 May 21
The present invention provides compositions such as aqueous liquid compositions and lyophilized compositions comprising a recombinant human acid sphingomyelinase.
Mark Yang, Claudia Buser, Bernardo Perez-Ramirez, Grant Trierweiler, Sangeeta Benjwal
Filed: 25 Nov 20
Utility
FC Containing Polypeptides with Altered Glycosylation and Reduced Effector Function
20 May 21
Provided are binding polypeptides (e.g., antibodies), and drug conjugates thereof, comprising an Fc domain with an altered glycosylation profile and reduced effector function.
Clark PAN, Huawei QIU
Filed: 8 Oct 20
Utility
Glucosylceramide synthase inhibitors
18 May 21
Elyse Bourque, Mario A. Cabrera-Salazar, Cassandra Celatka, Seng H. Cheng, Bradford Hirth, Andrew Good, Katherine Jancsics, John Marshall, Markus Metz, Ronald K. Scheule, Renato Skerlj, Yibin Xiang, Zhong Zhao, John Leonard, Thomas Natoli, Elina Makino, Herve Husson, Oxana Beskrovnaya
Filed: 18 Apr 17
Utility
Methods for Purification of Recombinant Aav Vectors
13 May 21
Provided herein are methods for the purification of recombinant adeno-associated virus (rAAV) vectors that can be used for gene transfer and specifically for gene therapy or vaccination.
Paulene McLean Quigley SHELDON, Peter S. GAGNON, Gina NICHOLS, Barbara A. THORNE
Filed: 28 Sep 20
Utility
Site-specific glycoengineering of targeting moieties
4 May 21
The current disclosure provides binding polypeptides (e.g., antibodies), and targeting moiety conjugates thereof, comprising a site-specifically engineered glycan linkage within native or engineered glycans of the binding polypeptide.
Luis Avila, Clark Pan, Huawei Qiu, Qun Zhou
Filed: 18 Mar 15
Utility
Compositions and Methods for Treating Spinal Muscular Atrophy
29 Apr 21
The present provides methods for treating spinal muscular atrophy using a self-complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN.
Marco A. PASSINI, Lamya S. SHIHABUDDIN, Catherine R. O'RIORDAN, Seng H. CHENG
Filed: 25 Sep 20